Fibroblastoma is an uncommon type of bone marrow cancer that disrupts the body’s normal production of blood cells and causes extensive scarring in the bone marrow, leading to severe weakness.
One-third of patients have pre-existing anemia, and most patients will develop it for the duration of their illness. For patients with severe anemia, survival is shortened to only about two years. Even with mild anemia, the median survival was 4.9 years. A drug is urgently needed to treat anemia in these patients.
Pioneering MF drugs developed in the past decade, such as ruxolitinib, inhibit harmful JAK signaling, while momelotinib was the first to inhibit both JAK and ACVR1.
Is Magic Pill ready for myelofibrosis?
To learn more about this, the researchers analyzed momelotinib against a second drug, danazol, in symptomatic and anemic MF patients previously treated with standard care JAK inhibitor therapy. It enrolled 195 MF patients in 21 countries.
Of the enrolled participants, 130 received momelotinib and 65 danazol. Participants did not know until after 24 weeks which drug they were receiving, and those in the danazol group were allowed to switch to momelotinib at that time.
Significant improvements in symptoms, spleen size and measures of anemia were observed. Favorable safety and trends in overall survival have been noted.
Participants required fewer blood transfusions to replace red blood cells and demonstrated better levels of oxygen-carrying hemoglobin. The study enrolled participants entirely within the COVID timeframe, which is remarkable.
Momelotinib met all primary and secondary endpoints and over the short six-month period there was a trend toward an overall survival benefit, which is also notable. It can be used as a single pill in the treatment of myelofibrosis in the future.